Original Title: Arzneimittel-Richtlinie/Anlage XII: Satralizumab (Neuromyelitis-optica-Spektrum-Erkrankungen, Anti-Aquaporin-4-IgG-seropositiv, ≥ 12 Jahre)

Authors' objectives: The Federal Joint Committee [Gemeinsamer Bundesausschuss (G-BA)] has had the legal task of carrying out an (additional) benefit assessment for all newly approved drugs with new active ingredients immediately after market entry (§ 35a SGB V). The result of this assessment is the basis for deciding how much the statutory health insurance pays for a new drug with a new active ingredient. The G-BA was commissioned to carry out the benefit assessment through the Pharmaceuticals Market Reorganisation Act [Gesetz zur Neuordnung des Arzneimittelmarktes (AMNOG)]. In the context of the early benefit assessment of medicinal products containing new active substances, the following rules apply to orphan drugs: According to the legal requirements (§ 35a SGB V), the additional medical benefit of these drugs is already considered to be proven by the approval. The G-BA determines the extent of the additional benefit for orphan drugs that do not exceed a turnover of 50 million Euros in the last twelve calendar months, on the basis of the approval and the studies justifying the approval.

Authors' results and conclusions: Satralizumab is approved for the treatment of neuromyelitis optica spectrum disease (NMOSD) in adults and adolescents aged 12 years and older who are anti-aquaporin-4 antibody (AQP4-AK)-positive. Satralizumab can be used as monotherapy or in combination with oral corticosteroids, azathioprine or mycophenolate mofetil. The benefit assessment is based on the randomised, double-blind, multicentre, placebo-controlled studies SAkuraStar and SAkuraSky. The SAkuraStar investigate satralizumab as monotherapy compared to placebo in the treatment of adults with NMOSD. The SAkuraSky study investigate satralizumab in combination with baseline immunosuppressive therapy compared to placebo plus baseline immunosuppressive therapy in the treatment of adults and adolescents aged 12 years and older with NMOSD. Only the results of the AQP4-AK-positive patients of both studies are relevant for the benefit assessment. No conclusions can be drawn regarding the effects of satralizumab on mortality as no deaths occurred during the double-blind study periods. There was a statistically significant positive effect of satralizumab monotherapy on both protocol-defined relapse and EDSS progression compared to placebo. The observed effect on relapse is associated with an unclear risk of bias, while the risk of bias for the EDSS progression is considered high. For the combination therapy, a statistically significant difference in favour of satralizumab compared to placebo was shown for protocol-defined relapse, while the difference in EDSS progression did not reach statistical significance. The risk of bias of both results are considered to be high. Valid data on patient-reported morbidity endpoints or on quality of life could not be taken into account. With high risk of bias, no differences were found between treatments groups in severe and serious adverse events (AE) and AE leading to treatment discontinuation in both studies. In general, data is very limited for adolescents with NMOSD.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/715/#english

Year Published: 2022

URL for published report: https://www.g-ba.de/downloads/39-1464-5229/2022-01-06_AM-RL-XII_Satralizumab_D-693_EN.pdf

Requestor: The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/715/#nutzenbewertung

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Germany

Organisation Name: The Federal Joint Committee

Contact Address: Gutenbergstr. 13, 10587 Berlin, Germany

Contact Name: Fachberatung Medizin [Department of Medical Consultancy]

Contact Email: Fachberatung-Medizin@g-ba.de

Copyright: https://www.g-ba.de/sys/impressum/